2025 Sponsors & Exhibitors

Sciensus is a leader in integrated commercial services with 30 years’ experience in navigating thecomplex European healthcare ecosystem to maximise patient access to medicines, accelerate productlaunches and achieve full commercialisation. ​ ...

Our purpose UCB is driven to ensure that everyone can live the best life they can, as free as possible from the challenges and uncertainty of disease. This ambition fuels our purpose: creating value in the lives of the people we serve, now and into ...

Clinigen is the market leader in Managed Access Programs, enabling pharmaceutical and biotech companies to deliver pre-approval and unlicensed medicines to patients in need worldwide. With more than 35 years’ experience, over 300 active programs...

Experience that counts for more. Charles River Associates (CRA) is a leading global consulting firm that offers economic, financial, and strategic expertise to major law firms, corporations, accounting firms, and governments around the world. Our...

Norgine is a mid-sized EU-based pharmaceutical company with 1,500 employees, generating approximately $600 million in annual sales. At Norgine, innovation drives our mission to deliver medicines that change lives. From common conditions like...

Parexel is among the world’s largest clinical research organizations (CROs), providing the full range of Phase I to IV clinical development services to help life-saving treatments reach patients faster. Leveraging the breadth of our...

Founded in 1997, Ergomed is a full-service, global Phase I-IV clinical development and trial management service partner with leading expertise in developing oncology and rare disease drugs.  Our portfolio includes experience working with novel...

As a business unit of RTI International, we provide global biopharmaceutical companies research and consulting services in health economics, patient-centered outcomes research, market access, and pharmacoepidemiology. As the only...

Sanofi is an innovative global healthcare company, driven by one purpose: to chase chasing the miracles of science to improve the health and quality of life of people around the world. Sanofi’extensive geographic footprint, operating  across nearly...

Volv Global is a pioneering AI-driven healthcare intelligence company, delivering unprecedented insights into rare and difficult-to-diagnose diseases. By leveraging population-scale data, Volv helps clinicians and pharmaceutical innovators recognize ...

Worldwide Clinical Trials (Worldwide) is a full-service global contract research organization (CRO) that works in partnership with biotechnology and pharmaceutical companies to create customized solutions that advance new medications – from...

Allucent is on a mission to help bring new therapies to light by solving the distinct challenges of small and mid-sized biotech companies. We’re a global provider of comprehensive drug development solutions, including regulatory consulting, clinical ...

Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities.

Rare Patient Voice, LLC provides patients and family caregivers an opportunity to participate in all types of healthcare research, including market research, health economics outcomes and real-world evidence, user experience/human factors...

SGS Integrated CDMO Solutions is a trusted partner in comprehensive, end-to-end pharmaceutical development and manufacturing services. With expertise spanning the entire drug lifecycle—from early development to full commercial supply—we help biotech ...

Valid Insight, part of the Bioscript Group, helps life science companies confidently navigate complex market access challenges. We specialise in strategic market access consulting across pharmaceuticals, medical nutrition, diagnostics, and medical...

Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is led by a team of highly experienced...

EveryONE Medicines is pioneering a scalable, global framework to design, develop, and manufacture individualized medicines for children with life-threatening neurodegenerative diseases. We are building an integrated, end-to-end system that leverages ...

IQVIA is a leading global provider of clinical research services, commercial insights and healthcare intelligence to the life sciences and healthcare industries. IQVIA’s portfolio of solutions are powered by IQVIA Connected Intelligence™ to deliver...

Sharp Therapeutics is a pre-clinical stage company developing first-choice small-molecule therapeutics for hereditary disorders. The Company's discovery platform combines novel high throughput screening technologies, with compound libraries...

Founded in 1997, ACA Pharma is a U.S.-based pharmaceutical distributor providing end-to-end commercialization for U.S. and European originators across Greater China and Southeast Asia. ACA specializes in fast-track regulatory pathways, market...

Aixial is a global CRO who have been conducting rare disease and orphan drug clinical trials since 2006.  With deep expertise and a wealth of experience Aixial is well placed to support pharmaceutical and biotech companies by offering a...

The Ataxia Charlevoix-Saguenay Foundation is a federally registered charity founded in 2006 in Montreal by parents of two children affected by Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS). Its mission is to discover and develop ...

Your global medicines access and clinical trial supplies solution partner. BAP Pharma specialises in providing tailored early and managed access solutions to HCP’s and patients across the globe, the development and production of clinical secondary...

Decisive Consulting Ltd. is a leading consulting firm specializing in providing strategic guidance, advocacy, and partnership opportunities for companies in the orphan drug industry. With a proven track record of success, they offer tailored...

FH Europe Foundation (FHEF) represents the needs of people with familial hyperlipidaemias. Covering both the most common genetic and inherited conditions in the world and the rare conditions, the Foundation acts in the area of advocacy and patient...

Genvade Therapeutics: Pioneering Therapies for Nonsense Mutation DisordersGenvade Therapeutics is a biotechnology start-up dedicated to developing therapies for patients with genetic diseases caused by nonsense mutations—mutations that account for...

GNH USA delivers global end-to-end integrated solutions for orphan drug development and access.From R&D to delivery, we support clinical trials, name patient supply, validated cold chain shipments, and compliant global distribution.With decades...

Regulatory Affairs Advice, Strategy, and Support Services for biopharma and medtech companies. The IDEA team are passionate about supporting patients with rare diseases and accelerating the path to market for orphan medicines by applying regulatory ...

As the leading kidney research charity in the UK, nothing is going to stop us in our urgent mission to end kidney disease. We’re here to be heard, to make a difference, to change the future. This is a disease that ruins and destroys lives. It must...

Medscape Education (medscape.org) is the leading destination for continuous professional development, consisting of more than 30 specialty-focused destinations offering thousands of free CME and CE courses and other educational programs for...

Onyx Scientific, the CDMO division of Ipca Laboratories, has specialized in small molecule drug substance development and manufacturing for 25 years. From our MHRA-licensed facilities in the UK, we provide seamless support for early development for...

Proteintech is a leading manufacturer of antibodies, immunoassays, nanobodies, and proteins. With over 300,000 product citations, Proteintech’s comprehensive product portfolio offers antibodies covering 2/3 of the human proteome, all manufactured...

Orphan drug developers need safe and effective advanced therapies with minimal off-target effects. Annogen offers validated, bespoke gene promoters —the genetic switches and genomic sites—to achieve highly specific gene expression in the right...

Azur Health Science, your premium medical writing and consulting agency, experts in rare disease With a team of expert writers, Azur Health Science offers a full-service regulatory writing consultancy in rare disease and precision...

BioMe, Inc. is a pre-clinical stage biotechnology company dedicated to transforming human healthcare through microbiome-based therapeutics. Leveraging its proprietary SURE™ platform, BioMe establishes clear disease–microbiome connections and...

Cellbyte supports global and local Commercial Pharma teams in the successful launch of new medicines. Its AI-based platform enables users to automate workflows across regulatory data, HTA assessments, and pricing information, or extract targeted...

CureAge therapeuctis is a biotech company developing genetic medicines for peripheral nerve diseases arising from Schwann cell dysfunction, starting with Neurofibromatosis Type 1 (NF1), a currently incurable rare disorder affecting more than twice...

Genvade Therapeutics: Pioneering Therapies for Nonsense Mutation Disorders Genvade Therapeutics is a biotechnology start-up dedicated to developing therapies for patients with genetic diseases caused by nonsense mutations—mutations that account...

iollo is a Stanford spinout, co-founded with Prof. Michael Snyder and backed by Y Combinator. We developed Quinn, an AI scientist built to rapidly accelerate pharma and biotech discovery.Quinn quickly integrates and analyzes complex multi-omics and...

Phenotypic screening is a versatile and impactful tool in the quest to develop therapies for rare disorders. By leveraging unbiased, functional approaches in disease-relevant cellular systems, this method overcomes many of the limitations of...

Mirava Bio is a BioCommercial Partner supporting emerging rare disease biotechs in defining the optimal cross-functional go-to-market strategy in EMEE and executing it through our fully licence-ready, plug-and-play commercialization...

NanoZymeX is a nanobiotechnology startup developing next-generation enzyme replacement therapies using proprietary lipid-based nanoparticles. Originating from research at the University of Basel, NanoZymeX integrates nanotechnology and protein...

Netabolics is a pioneering biotech company specializing in AI-driven biosimulation to decode the complexities of human biology. By integrating computational dynamical modeling with multiomics data, Netabolics enables precise identification and...

NeuroCores, Inc. is a preclinical biotech company, based in Boston, MA. The company's lead asset is KIT-13, a novel oral small molecule drug targeting Rett Syndrome, a rare pediatric neurodevelopmental disorder.NeuroCores is developing...

Olvia Biopharma helps emerging and mid-sized pharmaceutical and biotech companies expand globally through managed partnerships. Acting as your outsourced international business department, we design the right go-to-market model for each product and...

The Orphanet-AISBL is a newly established non-profit spinoff from Orphanet, dedicated to turning the more than five million combined data from the Orphanet Databse into actionable knowledge, thereby serving all stakeholders in the rare disease...

PharmaKrysto is a biopharma company focusd on developing new treatments for patients living with rare kidney disease.  Our lead programme is a small-molecule solution to the frequent formation of kidney stones in people with cystinuria. 

Scarlet Therapeutics is developing a unique platform that makes highly universal red blood cells from immortalised cell lines.  This allows scalable, and reporducible manufacture of modified or unmodified RBC for transfusion or disease treatment. ...

Senovia Biosciences is pioneering a new class of medicines built on a simple idea: the ketogenic diet works against seizures, but it’s impossible to live on as medicine. We’ve captured the power of ketosis in a drug. Our lead...

Trialize cuts the biggest operational pain‑points in clinical trials It turns the slowest, most error‑prone parts of trial operations into automated, real‑time workflows — compressing timelines and boosting data quality in one step.   Start‑up...

Unboxed Psychology is a behaviour science consultancy specialising in health psychology and behaviour change principles. Providing strategic services across healthcare working with pharmaceutical and biotech companies to infuse behaviour science...